March 20, 2024
Institutional Review Board, Hospital for Special Surgery
The safety of our participants is our top priority. The trial/study is approved and periodically reviewed by the Institutional Review Board (IRB), which includes doctors, administrators, ethicists, and members of the general public. The safety of clinical trials is reviewed by the U.S. Food and Drug Administration.
Before enrolling in a clinical trial or research study, the investigator will explain the purpose of the trial/study, its expected benefits, any possible risks or side effects, and what your role will be. If you want to join the trial/study, you must sign informed consent documents. You can leave a trial/study at any time without penalty.
For further information, see Understanding Clinical Trials/Research Studies.
Kyriakos A. Kirou, MD, DSc, FACP
David R. Fernandez, MD, PhD
Romy Kallas, MD
Tiredness is one of the most common conditions from persons that suffer from SLE. Based on initial research, it is believed that the antioxidant N-acetylcysteine (NAC) may improve tiredness by increasing the levels of the substance glutathione, which has been found to be lacking in people with SLE. NAC protects cells from poisonous body-damaging chemicals. The lack of glutathione (GSH) contributes to the dysfunction of the immune system which is damaging in patients with SLE and other autoimmune diseases. In earlier studies done in animals and human subjects, this medicine has been shown to increase the levels of glutathione in the blood. Bringing glutathione levels into a normal range may also improve overall disease outcome of SLE.
The purpose of this study is to find out the medical effectiveness of NAC during the course of a year. After an initial three-month open label period where eligible subjects all receive the study drug, those able to tolerate NAC will be randomized to receive either the study drug or a placebo (a sugar pill [inactive substance]) for the remainder of the trial. Some visits will involve blood draws and answering questionnaires (a list of questions). The blood you donate during the study visits will be used to see whether the levels of glutathione are raised and if there is an improvement in immune cell function because of taking NAC.
It is anticipated that 5 patients will be enrolled at HSS and a total of 230 people across all study sites with SLE is expected to be enrolled for this phase of the study.
1. ≥18 years of age
2. Fulfills ACR SLE classification criteria
3. Must have positive ANA at a titer of at least 1/80
4. Fulfills either BILAG 2004 index level A disease activity in at least 1 organ/system except renal or CNS, or BILAG 2004 index level B disease activity in at least 2 organs/systems if no level A disease activity is present AND SLEDAI of at least 6
5. Must be on stable immunosuppressants and/or antimalarials for 30 days prior to screening, stable oral corticosteroids for 2 weeks prior to screening, at most 20 mg/day prednisone or equivalent, stable belimumab or anifrolumab for 90 days prior to screening
6. Cannot be pregnant or lactating or planning to become pregnant
7. No cyclophosphamide within 3 months of screening
8. No rituximab within 12 months of screening or other biologic therapy within five half lives
9. No mTOR inhibitors (rapamycin/ sirolimus, everolimus)
Emily Wu, Research Coordinator
wue@HSS.EDU
212-774-2967